Sangamo and Pfizer collaborate to develop zinc finger protein gene therapy for ALS7 Jan 2018
"The precision and flexibility of zinc finger proteins enables targeting of virtually any genetic mutation."
Sangamo Therapeutics has announced a collaboration for the development of a potential gene therapy using zinc finger protein transcription factors (ZFP-TFs) to treat amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD) linked to mutations of the C9ORF72 gene.
ALS and FTLD are part of a spectrum of neurodegenerative disorders caused by mutations in the C9ORF72 gene that involve hundreds of additional repetitions of a six base pair sequence of DNA. This ultimately leads to the deterioration of motor neurons, in the case of ALS, or neurons in the frontal and temporal lobes, in the case of FTLD. Currently, there are no cures to halt or reverse the progression of ALS or FTLD. The C9ORF72 mutation is linked to approximately one-third of cases of familial ALS.
"We are excited to continue our collaborative relationship with Pfizer with this new program using Sangamo's zinc finger protein technology to develop a potential gene therapy for patients with certain forms of ALS and FTLD, devastating diseases with very limited treatment options," said Dr Sandy Macrae, CEO of Sangamo. "The precision and flexibility of zinc finger proteins enables targeting of virtually any genetic mutation. Collaboration with the right partner for a given therapeutic application is a key component of our corporate strategy and enables us to pursue the vast opportunity set of our platform."
"We look forward to working with Sangamo on potential treatments for devastating diseases related to genetic mutations of the C9ORF72 gene," said Greg LaRosa, Senior Vice President and Chief Scientific Officer, Pfizer Rare Disease. "Pfizer is proud of the progress we have made in the area of gene therapy, which offers tremendous promise to patients and their families."
Gene therapies are a potentially transformational technology for patients, focused on highly specialized, one-time treatments that address the root cause of diseases caused by genetic mutation. Sangamo's ZFP-TF technology involves introducing an engineered zinc finger protein (ZFP) which is designed to identify and bind to a precise sequence of DNA. Once bound to the target sequence of DNA, a transcriptional repressor domain attached to the ZFP suppresses expression of the gene. Under this collaboration, Sangamo and Pfizer will investigate allele-specific ZFP-TFs with the potential to differentiate the mutant C9ORF72 allele from the wild type allele and to specifically down-regulate expression of the mutant form of the gene.
Under the terms of the collaboration agreement, Sangamo will receive a $12 million upfront payment from Pfizer. Sangamo will be responsible for the development of ZFP-TF candidates. Pfizer will be operationally and financially responsible for subsequent research, development, manufacturing and commercialization for the C9ORF72 ZFP-TF program and any resulting products. Sangamo is eligible to receive potential development and commercial milestone payments of up to $150 million, as well as tiered royalties on net sales.
In May 2017, Sangamo and Pfizer entered into an exclusive, global collaboration and license agreement for the development and commercialization of potential gene therapy products for Hemophilia A, including SB-525, which entered the clinic in August 2017.
Natural lipid acts as potent anti-inflammatory
10 Jul 2018
Scientists see therapeutic potential against bacteria, viruses.Read more
Plasticell leads gene therapy manufacturing consortium
3 Jul 2018
Consortium to develop advanced technologies for the manufacturing of ex vivo gene therapies.Read more
Follicum develops new direct-to-scalp formulation
27 Jun 2018
The topical treatment for hair loss boasts a 'commercial' formulation.Read more
Collaboration to provide GMP-based services
19 Jun 2018
Researchers to benefit by receiving a rapid and cost-effective scaled supply of new compounds.Read more
Locate Therapeutics secures £2 million to fast-track development of innovative cell therapy programs
5 Jun 2018
TAOS matrix system and products hailed as potential ‘game changers’ in the field of regenerative medicine.Read more
Anti-vedolizumab antibodies to support treatment research
25 May 2018
A reliable source of antibodies for the development of bioanalytical assays for vedolizumab, to support research into the treatment of ulcerative colitis and Crohn’s diseaseRead more
The shortest way from the lab to continuous production
24 May 2018
Bosch's new Xelum R&D for continuous production of oral solid dosage forms ensures a short time-to-market and optimum dosing of APIs.Read more
Comparative analysis of a complex monoclonal antibody
24 May 2018
New poster describes how the EAF4 module enabled simultaneous measurement of size and surface charge characteristics of antibodies and proteins.Read more
Lilly expands oncology pipeline with acquisition
16 May 2018
Acquisition of AurKa Pharma expands Lilly's oncology pipeline with early-phase asset being studied in multiple tumour types.Read more
Versatile lyophilizer for research, development or small-scale production
15 May 2018
Allows for easy and intuitive scale-up from research.Read more
Are you a supplier
Here's what we can do for you
- Generate quality leads for your business
- Stay visible for 365 days of the year
- Receive product inquiries and respond to meeting requests directly
- Improve company online presence through Search Engine Optimisation